Bioclinica - Rare Diseasehttp://bioclinica.com/blog-categories/rare-disease
enRare Disease New Developmentshttp://bioclinica.com/blog/rare-disease-new-developments
<div class="field field-name-body field-type-text-with-summary field-label-hidden"><div class="field-items"><div class="field-item even" property="content:encoded"><p>As a follow-up to my previous blog post on <a href="/blog/growing-interest-rare-disease-pharma-and-biotech-industry" target="_self" title="rare disease development in the pharmaceutical and biotech industry">rare disease development in the pharmaceutical and biotech industry</a>, here's a chart to show the increase in interest in applications since 1983, which was taken from the FDA website.</p><div style="margin: 0 auto;"><table style="width: 500px;" align="center" border="1" cellpadding="5" cellspacing="0"><tbody><tr bgcolor="#BEBFB9"><th scope="col">Year</th><th scope="col">No. Approved</th><th scope="col">No. Designated</th><th scope="col">No. Designation Apps</th></tr><tr><td><div align="center">1983</div></td><td><div align="center">2</div></td><td><div align="center">1</div></td><td><div align="center">16</div></td></tr><tr><td><div align="center">1984</div></td><td><div align="center">3</div></td><td><div align="center">41</div></td><td><div align="center">48</div></td></tr><tr><td><div align="center">1985</div></td><td><div align="center">6</div></td><td><div align="center">48</div></td><td><div align="center">71</div></td></tr><tr><td><div align="center">1986</div></td><td><div align="center">6</div></td><td><div align="center">32</div></td><td><div align="center">58</div></td></tr><tr><td><div align="center">1987</div></td><td><div align="center">9</div></td><td><div align="center">58</div></td><td><div align="center">91</div></td></tr><tr><td><div align="center">1988</div></td><td><div align="center">10</div></td><td><div align="center">73</div></td><td><div align="center">88</div></td></tr><tr><td><div align="center">1989</div></td><td><div align="center">12</div></td><td><div align="center">76</div></td><td><div align="center">91</div></td></tr><tr><td><div align="center">1990</div></td><td><div align="center">12</div></td><td><div align="center">88</div></td><td><div align="center">131</div></td></tr><tr><td><div align="center">1991</div></td><td><div align="center">12</div></td><td><div align="center">79</div></td><td><div align="center">84</div></td></tr><tr><td><div align="center">1992</div></td><td><div align="center">14</div></td><td><div align="center">56</div></td><td><div align="center">77</div></td></tr><tr><td><div align="center">1993</div></td><td><div align="center">13</div></td><td><div align="center">62</div></td><td><div align="center">72</div></td></tr><tr><td><div align="center">1994</div></td><td><div align="center">11</div></td><td><div align="center">58</div></td><td><div align="center">82</div></td></tr><tr><td><div align="center">1995</div></td><td><div align="center">12</div></td><td><div align="center">55</div></td><td><div align="center">73</div></td></tr><tr><td><div align="center">1996</div></td><td><div align="center">25</div></td><td><div align="center">57</div></td><td><div align="center">77</div></td></tr><tr><td><div align="center">1997</div></td><td><div align="center">18</div></td><td><div align="center">51</div></td><td><div align="center">72</div></td></tr><tr><td><div align="center">1998</div></td><td><div align="center">20</div></td><td><div align="center">68</div></td><td><div align="center">125</div></td></tr><tr><td><div align="center">1999</div></td><td><div align="center">20</div></td><td><div align="center">74</div></td><td><div align="center">94</div></td></tr><tr><td><div align="center">2000</div></td><td><div align="center">13</div></td><td><div align="center">69</div></td><td><div align="center">88</div></td></tr><tr><td><div align="center">2001</div></td><td><div align="center">6</div></td><td><div align="center">76</div></td><td><div align="center">129</div></td></tr><tr><td><div align="center">2002</div></td><td><div align="center">14</div></td><td><div align="center">62</div></td><td><div align="center">121</div></td></tr><tr><td><div align="center">2003</div></td><td><div align="center">12</div></td><td><div align="center">95</div></td><td><div align="center">168</div></td></tr><tr><td><div align="center">2004</div></td><td><div align="center">14</div></td><td><div align="center">130</div></td><td><div align="center">174</div></td></tr><tr><td><div align="center">2005</div></td><td><div align="center">20</div></td><td><div align="center">122</div></td><td><div align="center">175</div></td></tr><tr><td><div align="center">2006</div></td><td><div align="center">22</div></td><td><div align="center">141</div></td><td><div align="center">190</div></td></tr><tr><td><div align="center">2007</div></td><td><div align="center">15</div></td><td><div align="center">119</div></td><td><div align="center">200</div></td></tr><tr><td><div align="center">2008</div></td><td><div align="center">15</div></td><td><div align="center">165</div></td><td><div align="center">185</div></td></tr><tr><td><div align="center">2009</div></td><td><div align="center">17</div></td><td><div align="center">160</div></td><td><div align="center">250</div></td></tr><tr><td><div align="center">2010</div></td><td><div align="center">14</div></td><td><div align="center">192</div></td><td><div align="center">323</div></td></tr><tr><td><div align="center"><strong>TOTAL</strong></div></td><td><div align="center"><strong>367</strong></div></td><td><div align="center"><strong>2308</strong></div></td><td><div align="center"><strong>3353</strong></div></td></tr></tbody></table></div><h3>So… What's New with Rare "Orphan" Disease?</h3><p>Besides the growing interest in rare disease and the new law, here are other events happening that show the growing spotlight on this niche:</p><ul><li>In January 2011, the FDA launched a new orphan drug database which could encourage drug manufacturers to conduct additional studies on their already approved products to add rare disease indications. The first <a href="http://www.terrapinn.com/2011/world-orphan-drug-congress" target="_blank">World Orphan Drug Congress</a> was held in December (2010) in Geneva. It brought together hundreds of participants from across the industry.<sup>1</sup></li><li><a href="http://www.rarediseaseday.org/" target="_blank">International "Rare Disease" Day</a> began in 2008 and since then, on February 28 more than 40 countries worldwide will organize awareness-raising activities.<sup>2</sup></li><li>Since it is not necessarily easy to get <a href="http://blogs.wsj.com/health/2011/03/22/partnership-bears-some-early-fruit-for-rare-disease/?mod=google_news_blog" target="_blank">clinical trials going to treat rare disease</a>, companies are partnering with patient advocacy organizations to drive the trial.<sup>3</sup></li><li>BioClinica has been involved in a number of products in development for orphan indication. I have personally been involved in helping support a number of these fascinating but sad rare disease states. Finding a cure for an unmet medical need is always fulfilling whether it affects several million or just a few hundred individuals. We will continue to support these rare disease efforts.</li></ul><p>While we continue to do so, is there anything that you are involved in regarding rare disease – no matter how big or small? Please share your experiences with me.</p><p>1 World Orphan Drug Congress Website. <a href="http://www.terrapinn.com/2011/world-orphan-drug-congress" target="_blank">www.terrapinn.com/2011/world-orphan-drug-congress</a><br />2 Rare Disease Day Website. <a href="http://www.rarediseaseday.org" target="_blank">www.rarediseaseday.org</a><br />3 Marcus, Amy Dockser. "Partnership Bears Some Early Fruit for Rare Disease." <em>Wall Street Journal's Health Blog</em>. March 22, 2011. <a href="http://blogs.wsj.com/health/2011/03/22/partnership-bears-some-early-fruit-for-rare-disease/?mod=google_news_blog" target="_blank">blogs.wsj.com/health/2011/03/22/partnership-bears-some-early-fruit-for-rare-disease/?mod=google_news_blog</a></p></div></div></div><div class="field field-name-field-blog-category field-type-taxonomy-term-reference field-label-inline clearfix"><div class="field-label">Category:&nbsp;</div><div class="field-items"><div class="field-item even"><a href="/blog-categories/medical-imaging" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Medical Imaging</a></div><div class="field-item odd"><a href="/blog-categories/rare-disease" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Rare Disease</a></div></div></div>Fri, 03 Jun 2011 12:45:00 +0000Bioclinica174 at http://bioclinica.comhttp://bioclinica.com/blog/rare-disease-new-developments#commentsGrowing Interest in Rare Disease for Pharma and Biotech Industryhttp://bioclinica.com/blog/growing-interest-rare-disease-pharma-and-biotech-industry
<div class="field field-name-body field-type-text-with-summary field-label-hidden"><div class="field-items"><div class="field-item even" property="content:encoded"><p>At BioClinica, we are involved in a number of rare disease studies. This is a growing area of development for the pharmaceutical and biotech industry. FDA’s office of Orphan Product Development reports that they saw a record 323 orphan drug designation requests come through the door in 2010. It's unclear why – one reason could be OOPD's outreach efforts.</p><p><a href="http://en.wikipedia.org/wiki/Rare_disease" target="_blank">Rare diseases</a> are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in Europe. It is estimated that in the United States, 47% of rare disorders affect fewer than 25,000 people. Because patient populations for rare diseases are so small, big pharmaceutical companies historically have had little financial incentive to invest in drug development for these diseases.</p><p>More than 6,000 rare diseases have been identified as orphan illnesses. There are more than 2,000 patient organizations in existence that offer support to patients seeking help and answers regarding these rare conditions.</p><h3>US Orphan Drug Act of 1983 &amp; "Improving Access to Clinical Trials" Act</h3><p>The United States Orphan Drug Act of 1983 created tax incentives available for organizations that work on clinical trials for an orphan disease. It also gives the company drug exclusivity on the market for 7 years.</p><p>In October 2010, President Obama signed S. 1674 ("<a href="http://www.govtrack.us/congress/bill.xpd?bill=s111-1674" target="_blank">Improving Access to Clinical Trials Act</a>") into law. The law provides an exclusion under the Supplemental Security Income program and the Medicaid program for compensation to be provided to individuals who participate in <a href="/blog/how-time-quality-cost-metrics-affect-clinical-trials-and-programs">clinical trials</a> for rare diseases or conditions.</p><h3>Pharma Involvement in "Orphan" Drug Market</h3><p>In an October 2010 article in Life Science Leader titled, "<a href="http://www.lifescienceleader.com/index.php?option=com_jambozine&amp;layout=article&amp;view=page&amp;aid=4113" target="_blank">Orphan Drug: Big Pharma's Next Act?</a>," Cliff Minty, Ph.D. writes:</p><p>"<em>Pharmaceutical companies that have already successfully tested the orphan drug market include Novartis (Gleevac, Tasigna, and Ilaris), Novo Nordisk (Nordotropin and NovoSeven) and Eli Lilly (Humatrope). However, big pharma's interest in orphan drug development is clearly on the rise. Pfizer, the world’s largest drug maker, announced in July 2010 that it was creating a new division to focus exclusively on new treatments for rare diseases. Likewise, in February 2010, GlaxoSmithKline announced it was creating a dedicated unit specializing in orphan drug development. Other big pharma companies including Novartis and Eli Lilly have also invested in active orphan drug development programs.</em>"<sup>1</sup></p><h3>Why Discuss Rare Diseases?</h3><p>Because rare disease sufferers usually have severely limited treatment options, it is generally accepted that orphan drugs may have an advantage in garnering regulatory approval — as compared with drugs with non-orphan drug status — even though their safety profiles may be less than ideal.</p><p>Another point to note is that when a drug treats rare disease symptoms, it can be used to treat symptoms of other diseases as well.</p><p>Also, the knowledge that is gathered from researching rare diseases can have larger benefits. For example, scientists who studied Kuru, a incurable degenerative neurological disorder that is a type of transmissible spongiform encephalopathy, have led to a better understanding of similar, more common <a href="http://health.howstuffworks.com/diseases-conditions/rare/rare-diseases2.htm" target="_blank">prion diseases</a>, such as "Mad Cow" disease.<sup>2</sup></p><p>Part 2 of this series will go into more granularity of some of the current aspects of rare diseases.</p><p>Do you know of a rare disease in particular that – upon research – turned out to have larger benefits for society as a whole? Where do you see the pharma and biotech industries taking the new law regarding orphan disease research? Please share your comments with me below.</p><p><sup>1</sup>Mintz, Ph.D., Cliff. "Orphan Drugs: Big Pharma's Next Act?" <em>Life Science Leader.</em> October 2010. <a href="http://www.lifescienceleader.com/index.php?option=com_jambozine&amp;layout=article&amp;view=page&amp;aid=4113" target="_blank">www.lifescienceleader.com/index.php?option=com_jambozine&amp;layout=article&amp;view=page&amp;aid=4113</a></p><p><sup>2</sup>Lamb, Robert. "What are the world’s rarest diseases?" <em>Discovery Fit &amp; Health</em>. <a href="http://health.howstuffworks.com/diseases-conditions/rare/rare-diseases2.htm" target="_blank">health.howstuffworks.com/diseases-conditions/rare/rare-diseases2.htm</a></p></div></div></div><div class="field field-name-field-blog-category field-type-taxonomy-term-reference field-label-inline clearfix"><div class="field-label">Category:&nbsp;</div><div class="field-items"><div class="field-item even"><a href="/blog-categories/medical-imaging" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Medical Imaging</a></div><div class="field-item odd"><a href="/blog-categories/rare-disease" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Rare Disease</a></div></div></div>Wed, 01 Jun 2011 19:49:00 +0000Bioclinica176 at http://bioclinica.comhttp://bioclinica.com/blog/growing-interest-rare-disease-pharma-and-biotech-industry#comments